Over the past year, initiative named members, held two meetings, and gave out $9.4 million.

Early last year, leaders of the NIH and FDA announced that they would team up on issues of regulatory science as well as translational medicine. The agencies said their partnership was designed to “accelerate the process from scientific breakthrough to the availability of new, innovative medical therapies for patients.”

The leadership council’s task was to “spearhead collaborative work on important public health issues” by joining forces “to help ensure that regulatory considerations form an integral component of biomedical research planning, and that the latest science is integrated into the regulatory review process,” NIH and FDA noted.

What has happened since then? The partnership is alive and well, even if not exactly high-profile. The NIH and FDA did announce the awarding of four research grants totaling $9.4 million last September. In doling out this money, the agencies said they hoped to advance regulatory science, i.e., the development and use of the scientific knowledge, tools, standards, and approaches necessary for the assessment of medical product safety, efficacy, quality, potency, and performance.

Less visibly, but perhaps no less importantly, the agencies have named the members of the Joint NIH-FDA Leadership Council created to carry out the goals of the partnership. The council is co-chaired by the heads of both agencies, NIH director Francis S. Collins, M.D., Ph.D., and Commissioner of Food and Drugs Margaret A. Hamburg, M.D.

According to a transcript of a meeting held November 15, 2010, Jesse L. Goodman, M.D., chief scientist and deputy commissioner for science and public health, told the agency’s science board that the leadership council has set up working groups to identify several areas of opportunity or priorities for future efforts. Amy Patterson, M.D., NIH’s associate director of Science Policy, recently told GEN, “Through the working groups of the leadership council, systematic efforts are under way to address these areas and other high priority areas for collaboration.”

To date, the council has had two meetings: an organizing teleconference and a 2.5 hour in-person meeting. Two additional meetings are planned for later this year, Dr. Patterson said.

One priority Dr. Goodman identified as an area of interest for the council was to examine how NIH and FDA currently work together and find new ways to partner. While all of FDA’s centers interact with NIH, he said, the agencies should “really pick a few areas where, on levels across the entire NIH and across the entire FDA, we can really make a difference working together.

“An example we’ve discussed a lot is earlier engagement with some of their clinical enterprise studies, so that the results are as useful as possible at the end of the day in impacting medical care and improving the lives of patients,” Dr. Goodman told the FDA’s science board.

Other priorities for the council include:

• Optimizing and maximizing data from clinical trials

• Encouraging the repurposing of products not being used or which have dropped through the commercial development system, such as the repurposing of drugs for orphan diseases

• Developing science to back up the work that the Center for Tobacco Products is going to do: The center’s duties include reviewing premarket applications for new and modified-risk tobacco products, requiring new warning labels, and establishing and enforcing advertising and promotion restrictions.

“I think it’s sort of astonishing, beyond kind of the basic science of harm, how little science there is about tobacco and its effects. We have heard from Dr. Deyton, the new director of the Center, how important it’s going to be for them as they start getting, for example, lists of ingredients and start trying to find effective ways to reduce harm, to have the science to back it up,” Dr. Goodman told the board.

FDA science board member Garret Fitzgerald, M.D., suggested to the leadership council another area where NIH-FDA cooperation could prove valuable: Applying the convergence of new technologies such as next-generation sequencing, medicalomics, and microbiomics to engage new researchers in food science as well as “provide new tools to those who are interested to begin to ask questions with a degree of precision that was previously unimaginable.”

Already in the Works

Also discussed before the FDA’s Science Board was the $9.4 million in grants awarded over three years to support four research projects in regulatory science. Of that total, approximately $950,000 came from FDA and the rest from NIH.

The grant winners and their projects are:

• William G. Barsan, M.D., Donald A. Berry, Ph.D., and Roger J. Lewis, M.D., Ph.D., University of Michigan, Ann Arbor: identifying effective approaches including statistical methods and organizational processes for design and conduct of high-quality, confirmatory, adaptive clinical trials to accelerate the development and evaluation of promising new therapies and clinical devices

• Daniel R. Cerven and George L. DeGeorge, Ph.D., MB Research Laboratories: developing and conducting prevalidation testing of the Replacement Ocular Battery (ROBatt). It consists of four alternative ocular irritancy assays that may eventually lead to replacement of rabbits used in existing regulatory ocular safety assessments of consumer products, chemicals, and raw materials

• Dennis E. Hourcade, Ph.D., Washington University, St. Louis: developing a new method to predict the potential harmful effects of nanoparticles intended for use in clinical applications prior to their testing in humans

• Donald E. Ingber, M.D., Ph.D., Harvard University Medical School, Boston: building a new “heart-lung micromachine” composed of microfluidic channels lined by living cells for use as a safety and efficacy screening platform that could eventually replace animal assays currently used for development and regulatory review of drugs and nanotherapies.

“NIH support for this area of research is expected to continue and to be an important activity of the new National Center for Advancing Translational Sciences (NCATS),” Dr. Patterson said. NIH plans to launch the $700 million NCATS in October, with the goal of accelerating translation of basic research discoveries into new drugs and devices.

More relevant to the NIH-FDA partnership, Dr. Collins said in NIH’s presentation of its fiscal year 2012 budget request, NCATS will also “facilitate the regulatory review process through recent initiatives including the NIH-FDA Leadership Council and a research program in regulatory science.”

NCATS is envisioned as part of NIH’s FY 2012 budget, for which President Obama has requested a 2.4%—or $745 million—spending increase for the agency, to $31.987 billion. President Obama on Friday signed a continuing resolution that cuts NIH funding by $260 million for 2011. The cut is a compromise between the Republican-controlled House of Representatives, which proposed cutting NIH funding by $1.6 billion, and the Democratic-controlled Senate, which sought to freeze NIH spending at the FY 2010 level.